Outcomes of progranulin gene therapy in the retina are dependent on time and route of delivery.

Mol Ther Methods Clin Dev · 2021 · PMC8390452 · PMID 34485593

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showed a trendno p-value reported

PGRN −/− retinas showed a trend in lipofuscin increase when compared to C57BL/6J retinas at months 1, 3, and 9.

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a slight trendno p-value reported

While cohorts treated at 1 and 6 months of age showed a slight trend in retinal thinning when they expressed progranulin in comparison to contralateral control eyes ( Figures 3 A–3C), animals injected at 12 months of age exhibited statistically significant reductions in retinal thickness ( Figure 4 I).

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